Herzeg, Akos;
Almeida-Porada, Graca;
Charo, R Alta;
David, Anna L;
Gonzalez-Velez, Juan;
Gupta, Nalin;
Lapteva, Larissa;
... MacKenzie, Tippi C; + view all
(2022)
Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting).
Journal of Clinical Pharmacology
, 62
(S1)
S36-S52.
10.1002/jcph.2127.
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Abstract
We are living in a golden age of medicine in which the availability of prenatal diagnosis, fetal therapy, and gene therapy/editing make it theoretically possible to repair almost any defect in the genetic code. Furthermore, the ability to diagnose genetic disorders before birth and the presence of established surgical techniques enable these therapies to be delivered safely to the fetus. Prenatal therapies are generally used in the second or early third trimester for severe, life-threatening disorders for which there is a clear rationale for intervening before birth. While there has been promising work for prenatal gene therapy in preclinical models, the path to a clinical prenatal gene therapy approach is complex. We recently held a conference with the University of California, San Francisco-Stanford Center of Excellence in Regulatory Science and Innovation, researchers, patient advocates, regulatory (members of the Food and Drug Administration), and other stakeholders to review the scientific background and rationale for prenatal somatic cell gene therapy for severe monogenic diseases and initiate a dialogue toward a safe regulatory path for phase 1 clinical trials. This review represents a summary of the considerations and discussions from these conversations.
| Type: | Article |
|---|---|
| Title: | Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting) |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1002/jcph.2127 |
| Publisher version: | https://doi.org/10.1002/jcph.2127 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Science & Technology, Life Sciences & Biomedicine, Pharmacology & Pharmacy, drug development, fetal medicine, immunopharmacology, neurology, pediatrics, perinatology, pharmacogenetics, pharmacogenomics, rare diseases, regulatory, scientific affairs, women's health, MOLONEY LEUKEMIA-VIRUS, INTRAUTERINE BLOOD-TRANSFUSION, IN-UTERO TRANSFER, GERM-LINE, LENTIVIRAL VECTORS, DIRECT-INJECTION, GESTATIONAL-AGE, SHAM CONTROL, T-CELLS, EXPRESSION |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL EGA Institute for Womens Health > Maternal and Fetal Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL EGA Institute for Womens Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10156950 |
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